Minimally invasive skin biopsies may be used to diagnose transthyretin amyloidosis and guide treatment in people with the rare progressive disease, according to the findings…
hATTR-PN
The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a Phase 3 clinical trial testing the investigational gene-editing therapy, nexiguran ziclumeran…
More than half of adults with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), a condition marked by heart damage, also have multi-nerve damage, according to…
HATTR-PN
NewsMRI detects brain changes before hATTR-PN symptoms begin
MRI scans detect brain changes in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) before symptoms appear, according to a study. While these changes…
HATTR-PN
ColumnsA look at the emotional and psychological effects of diagnosis
A diagnosis of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) between the ages of 30 and 60 can be profoundly life-altering. The gravity of the…
Nerve ultrasound — a scan that uses high-frequency sound waves to create real-time images of the body’s nerves — may be used to monitor signs…
Adults who carry the Val30Met, or V30M, mutation — the most common cause of early-onset hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) — often develop heart…
HATTR-PN
ColumnsThe emotional burden of receiving negative genetic test results
Hereditary diseases not only affect the body, but they also ripple through families, shaping relationships, identities, and mental health. I know this reality all too…
RNA-targeted therapies — ones that zero in on molecules acting as messengers for genetic information in cells — are safe and effective at slowing the…
HATTR-PN
NewsHereditary ATTR presents with more severe nerve damage
Polyneuropathy, or widespread nerve damage, occurs in both hereditary and wild-type transthyretin amyloidosis (ATTR), but the hereditary form, which includes familial amyloid polyneuropathy (FAP),…
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